Cellatica

Researchers are evaluating the effectiveness and safety of empasiprubart compared to intravenous immunoglobulin (IVIg) in adults diagnosed with Multifocal Motor Neuropathy (MMN). This Phase 3 study includes a double-blinded phase where participants receive either empasiprubart or IVIg, followed by an open-label phase where all receive empasiprubart. The study aims to assess treatment impact over a maximum duration of up to 49 months. Participants receive intravenous infusions of either empasiprubart or IVIg during the double-blinded phase, with matching placebo infusions given to maintain blinding. In the open-label phase, all participants are given empasiprubart infusions. The study carefully monitors treatments and their effects throughout these periods. Participants undergo assessments including measuring changes in grip strength of their most affected hand, using a 3-day moving average at week 24 as the primary outcome. Safety and efficacy are tracked throughout the study duration, with detailed follow-up visits and evaluations to monitor participant response and well-being during the study period.

Researchers are evaluating the efficacy of claseprubart (DNTH103) compared to placebo in adults with chronic inflammatory demyelinating polyneuropathy (CIDP) in this Phase 3 study. The goal is to assess how well claseprubart works in treating this condition, which involves nerve inflammation leading to muscle weakness and sensory problems. The study consists of multiple periods: Part A is an open-label phase lasting up to 13 weeks where all participants receive claseprubart. Those who respond move to Part B, a randomized, double-blind, placebo-controlled phase lasting up to 52 weeks, where participants receive either claseprubart or placebo by infusion or injection. After Part B, eligible participants may join an optional open-label extension for up to 104 weeks. A safety follow-up period of 40 weeks follows the treatment phases. Participants will undergo various assessments including neurological evaluations and disease activity scoring. Researchers will monitor the time from the first dose to disease relapse as the main outcome. Additional safety and efficacy measures will be tracked throughout all study periods. Total participation may last over two years including extension and follow-up phases.

Researchers are evaluating the safety and effectiveness of masitinib combined with riluzole compared to a placebo combined with riluzole for treating patients with Amyotrophic Lateral Sclerosis (ALS). Masitinib is an oral drug that targets specific cells involved in neuroinflammation, such as mast cells and microglia, which are believed to play a key role in ALS progression. This Phase 3 study is designed to better understand how masitinib may affect the disease by slowing progression and reducing inflammation in the nervous system. The study is a multicenter, double-blind, randomized, placebo-controlled trial where participants receive either oral masitinib at doses titrated to 4.5 or 6.0 mg/kg/day plus riluzole, or a matching placebo plus riluzole. Riluzole is given as a 50 mg tablet taken by mouth. The treatment period includes two ascending dose titrations for masitinib or placebo groups to compare outcomes and safety. Participants continue their stable riluzole treatment throughout the study. During the trial, participants are monitored for changes in their ALS Functional Rating Scale-Revised (ALSFRS-R) scores over 48 weeks, which measures their physical function and disease progression. Assessments include clinical evaluations and safety monitoring to track the impact of the treatments. The study enrolls adults aged 18 to 81 years who have probable or definite ALS and meet specified disease duration and function criteria. The goal is to gather detailed information on how masitinib in combination with riluzole might alter the course of ALS compared to riluzole alone.

The Pompe Registry is a global, multicenter, international program that follows patients with Pompe disease over time. It is an observational and voluntary study designed to track the natural history and outcomes of Pompe disease in both treated and untreated patients. The registry aims to improve understanding of the disease's variability, progression, identification, and natural history, with the goal of guiding and assessing therapeutic interventions. It also supports the Pompe medical community in developing monitoring recommendations and reporting patient outcomes to optimize care. Additionally, the registry helps characterize the Pompe disease population and evaluates the long-term effectiveness of alglucosidase alfa. This study collects data retrospectively and prospectively from patients worldwide diagnosed with Pompe disease. It does not involve any specific interventions or treatments but gathers comprehensive clinical information over time. Data collection includes medical history, diagnosis details, treatment status, and other relevant health information to better understand the disease and patient experiences. Participants contribute data through regular updates that capture their disease progression and treatment outcomes. Researchers use this information to study how Pompe disease manifests and changes over time, with a maximum follow-up period of 30 years. The registry helps fulfill regulatory commitments, supports product development and reimbursement, and provides valuable information for research and patient care improvements.